Investigational Drug (Encaleret) Restores Parathyroid Function in Rare Disease; Blood Calcium Levels Normalized in Clinical Trial Participants with ADH1

NIDCR Senior Research Physician, Rachel I. Gafni, MD, examines a clinical trial participant with ADH1, a rare genetic disorder marked by abnormally low levels of calcium in the blood. (Credit: NIDCR).
An investigational drug, encaleret, restored calcium levels in people with autosomal dominant hypocalcemia type 1 (ADH1), a rare genetic disorder marked by an imbalance of calcium in the blood and urine, as well as abnormally low levels of parathyroid hormone, which regulates blood calcium levels. Led by clinician-scientists from the National Institute of Dental and Craniofacial Research (NIDCR) at the National Institutes of Health’s Clinical Center, results from the clinical trial were published on September 28, 2023 in the New England Journal of Medicine. This article is titled Efficacy and Safety of Encaleret in Autosomal Dominant Hypocalcemia Type 1.”
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